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A new treatment for Alzheimer’s disease is able to safely and successfully lower levels of tau – a protein predominantly found in brain cells that is thought to be a cause of the disease.
The treatment works by injecting a drug (known as BIIB080) into the patient’s spinal canal. The drug then ‘silences’ the gene coding for the tau protein, which prevents the gene from being translated into the protein in a doseable and reversible way.
There are currently no treatments targeting tau, and the clinical trial, led by consultant neurologist Dr Catherine Mummery (UCL), is the first to take a ‘gene silencing’ approach to dementia and Alzheimer’s disease.
There are two other drugs approved for use by the FDA (aducanumab and lecanemab) but these treatments target a separate disease mechanism in Alzheimer’s disease – the accumulation of amyloid plaques.
Further trials will be need to be conducted in larger groups, the researchers say, but the phase 1 results show promise that this method is able to lower the production of harmful proteins and alter the course of Alzheimer’s disease.
What did the trial find?
The phase 1 trial, published in Nature Medicine, looked at the safety of BIIB080, what it does in the body, and how well it is able to ‘silence’ the gene coding for the tau protein.
In total, 46 patients, with an average age of 66, were enrolled in the trial. It took placed over a three-year period between 2017 and 2020 at the Leonard Wolfson Experimental Neurology Centre at NHNN.
The trial looked at three doses of the drug compared to placebo and found it to be well-tolerated, with all patients completing the treatment period and over 90% completing the post-treatment period.
No serious adverse effects were seen in patients given the drug, but some mild to moderate side effect were reported – the most common being a headache following the injection. However, these side effects were also reported in the placebo group.
The researchers also found that, after 24 weeks, the drug was able to reduce levels of total tau and phosphor tau concentration in the central nervous system by more than 50%.
Drug could slow or reverse Alzheimer’s disease
Dr Mummery says the results show promise for a potential Alzheimer’s treatment in the future, and more research will now need to be carried out to confirm the findings.
She said: “We will need further research to understand the extent to which the drug can slow progression of physical symptoms of disease and evaluate the drug in older and larger groups of people and in more diverse populations.
“But the results are a significant step forward in demonstrating that we can successfully target tau with a gene silencing drug to slow – or possibly even reverse – Alzheimer’s disease, and other diseases caused by tau accumulation in the future.